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Endosomolytic Agents for Gene Therapy
专利权人:
UCB Biopharma SPRL
发明人:
Terence Seward Baker,Michael Anthony William Eaton,Timothy John Norman,James Petrie Turner
申请号:
US16337243
公开号:
US20190298859A1
申请日:
2017.09.27
申请国别(地区):
US
年份:
2019
代理人:
摘要:
Compounds of formula (I), wherein Ar is an aryl group optionally further substituted with one or more groups R3; A is a lipophilic, hydrophobic moiety; R1 is a phosphodiester, phosphotriester, thioether or amide group; X is an unsubstituted or substituted C6 to C24 alkylene or alkenylene group, which is optionally interrupted by one or more —;NR9—;, —;O—; or —;S—; linkages, R2 is —;YC(R4)(R5)CO2R6; and pharmaceutically acceptable salts or solvates thereof are useful as endosomolytic agents particularly for the delivery of nucleic acids useful in gene therapy.
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